Aeglea BioTherapeutics Submits BLA to FDA for Pegzilarginase for the Treatment of Arginase 1 Deficiency SEE ORIGINAL STORY HERE BLA submission provides FDA with all pegzilarginase program data to review in detail; Aeglea looks forward to working collaboratively with the FDA on next steps in the review process If approved, pegzilarginase would be the [...]
REDACTED - FDA Family-Led Listening Session Report On June 10, 2021, a group of patients and families affected by Arginase 1 Deficiency (ARG1-D) participated in a one-hour virtual listening session with officials from the Food and Drug Administration (FDA). The session enabled those with ARG1-D and their caregivers to share their experiences, perspectives, needs, goals [...]
We’re proud to partner with the Child Neurology Foundation to conduct a survey to understand caregivers’ experiences on their journey in child neurology. Results will be shared with advocates and health care providers, so they are better able serve families. Caregivers of children with neurologic conditions can complete the brief survey thru April 1st at: www.surveymonkey.com/r/2022CNFCaregiverAssessment
PEGZILARGINASE Trial Discussion. Amazing presentation with Dr. Diaz, Dr. Burton and our own Tanja Brandt discuss Arginase 1 Deficiency, the Phase 2 and Phase 3 trial. Tanja discusses the day to day care taking of an individual with Arg1D. Great information on Arginase Deficiency 1 and the future of treating with the enzyme. This link [...]
I am excited to share with you that today we announced our Phase 3 PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints) study achieved its primary endpoint with a statistically significant reduction in plasma arginine levels. Pronounced and sustained plasma arginine reduction was accompanied by a positive trend in the Gross Motor Function Measure Part E (GMFM-E), a key clinical assessment of patient mobility. Based on these [...]
A Song for Jackson This song is dedicated to Jean, my partner, our son Jackson, the children and families affected by Arginase 1 Deficiency, Urea Cycle Defects, and rare diseases. My name is Leafy Wilson. In May of 2017, I started writing this song about the experience shared with my partner Jean, our son [...]
Meet Jackson Meet Jackson, age 30, with parents Jean and Leafy Southern California Living with ARG1-D Jackson, age 30, is a self-described cooking fanatic. It is a joyful family experience, involving his mom Jean and stepmom Leafy, bringing this already close-knit family even closer together. “I love to make Thanksgiving dinner, with all the [...]
Meet Nina Meet Nina At seven years old, Nina has never met a stranger – each person she meets is immediately her friend. Nina loves to paint, to create and would do arts and crafts all day if her parents would let her. “Nina simply has a way of making others feel good,” her [...]
FDA Family-Led Listening Session Report On June 10, 2021, a group of patients and families affected by Arginase 1 Deficiency (ARG1-D) participated in a one-hour virtual listening session with officials from the Food and Drug Administration (FDA). The session enabled those with ARG1-D and their caregivers to share their experiences, perspectives, needs, goals with officials [...]
Meet Willow Since the moment her mom Tanja realized she was pregnant, her daughter Willow has been full of surprises. Today, Willow is a nurturing, kind, sweet 10-year-old, who loves her new dog Teddy. She is also thoughtful, strong-willed, and stubborn – qualities she needs as she lives with Arginase 1 Deficiency (ARG1-D). Willow [...]
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